The Development and Pilot Testing of a Fidelity Checklist for a Family-Centered Telehealth Intervention for Parents of Children with Motor Delay.

This multi-methods study describes the development of a pediatric rehabilitation telehealth intervention fidelity checklist, estimates its inter-rater reliability, and documents raters' implementation experience. A literature scan and expert consultation identified eighteen key behaviors and categorized them into three subdomains, measured using a 5-point measurement system. To estimate the checklist's inter-rater reliability, three raters scored 33 video recordings. A Shrout and Fleiss Class 1,1 intraclass correlation (ICC)) and 95% confidence intervals (CI) calculated ICCs = 0.5 (CI: 0, 0.9) for both the Therapist and the Parent-Therapists subdomains, and the Parent subdomain an ICC = 0.3 (CI: 0, 0.8). In the implementation surveys, raters reported high levels of satisfaction (100%), ease of use (84% to 88%), and confidence in their video ratings (87% to 100%). Changes in procedures and scoring were recommended. Capturing raters' implementation experiences is crucial in the early evaluation of the fidelity checklists for telehealth.

Perceived strengths and weaknesses of paediatric physiotherapy services: Results from an international survey.

BACKGROUND AND PURPOSE: Ensuring access to high quality services in paediatric physiotherapy (PT) is important to respond to the diverse needs of children. The accessibility and quality of paediatric PT services has however never been explored internationally. The purpose of this study is to explore the perceived strengths, weaknesses, opportunities, and threats (SWOT) of paediatric PT services offered around the world. METHOD: A cross-sectional survey design method was used with a subsample of physiotherapists (PTs) who had previously participated in an online survey. The survey used for this study included close- and open-ended questions about access to services and the SWOT of PT services within participants' country. Descriptive statistics were used to summarize quantitative data and a content analysis was performed on open-ended questions. RESULTS: Overall, 47 PTs from 47 countries completed the survey; 36% of participants reported that free access was available to all children in their country while 34% stated that a referral was always required when accessing services. Lack of direct access, insufficient specialized PT, financial and geographical issues were the main perceived barriers to access services. Access also emerged as one of the nine themes following the SWOT analysis. Other themes included education, quality of PT approaches, PT practices, communication and cooperation, teamwork, government, resources, and attitudes of PTs. DISCUSSION: Despite variations in accessing services and how services are delivered across countries, some similar themes influencing PTs practices were found. Future opportunities for PTs working with children should aim at optimizing the initial training and professional development of PTs in paediatrics, increasing access to services for all children and advocating for sustainable and well-coordinated models of care building on best practices.

Training Intervention and Program of Support for Fostering the Adoption of Family-Centered Telehealth in Pediatric Rehabilitation: Protocol for a Multimethod, Prospective, Hybrid Type 3 Implementation-Effectiveness Study.

BACKGROUND: Children with disability face long wait times for rehabilitation services. Before the COVID-19 pandemic, telehealth adoption was low across pediatric rehabilitation. Owing to the COVID-19 pandemic restrictions, pediatric therapists were asked to rapidly shift to telehealth, often with minimal training. To facilitate the behavior changes necessary for telehealth adoption, provision of appropriate evidence-based training and support is required. However, evidence to support the effective implementation of such training is lacking. The successful real-world implementation of a training intervention and program of support (TIPS) targeting pediatric therapists to enhance the adoption of family-centered telerehabilitation (FCT) requires the evaluation of both implementation and effectiveness. OBJECTIVE: This study aimed to evaluate TIPS implementation in different pediatric rehabilitation settings and assess TIPS effectiveness, as it relates to therapists' adoption, service wait times, families' perception of service quality, and costs. METHODS: This 4-year, pan-Canadian study involves managers, pediatric occupational therapists, physiotherapists, speech-language pathologists, and families from 20 sites in 8 provincial jurisdictions. It will use a multimethod, prospective, hybrid type 3 implementation-effectiveness design. An interrupted time series will assess TIPS implementation. TIPS will comprise a 1-month training intervention with self-paced learning modules and a webinar, followed by an 11-month support program, including monthly site meetings and access to a virtual community of practice. Longitudinal mixed modeling will be used to analyze indicators of therapists' adoption of and fidelity to FCT collected at 10 time points. To identify barriers and facilitators to adoption and fidelity, qualitative data will be collected during implementation and analyzed using a deductive-inductive thematic approach. To evaluate effectiveness, a quasi-experimental pretest-posttest design will use questionnaires to evaluate TIPS effectiveness at service, therapist, and family levels. Generalized linear mixed effects models will be used in data analysis. Manager, therapist, and family interviews will be conducted after implementation and analyzed using reflective thematic analysis. Finally, cost data will be gathered to calculate public system and societal costs. RESULTS: Ethics approval has been obtained from 2 jurisdictions (February 2022 and July 2022); approval is pending in the others. In total, 20 sites have been recruited, and data collection is anticipated to start in September 2022 and is projected to be completed by September 2024. Data analysis will occur concurrently with data collection, with results disseminated throughout the study period. CONCLUSIONS: This study will generate knowledge about the effectiveness of TIPS targeting pediatric therapists to enhance FCT adoption in pediatric rehabilitation settings, identify facilitators for and barriers to adoption, and document the impact of telehealth adoption on therapists, services, and families. The study knowledge gained will refine the training intervention, enhance intervention uptake, and support the integration of telehealth as a consistent pediatric rehabilitation service option for families of children with disabilities. TRIAL REGISTRATION: ClinicalTrials.gov NCT05312827; https://clinicaltrials.gov/ct2/show/NCT05312827. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/40218.

Scope and Practices of Physical Therapists Working With Children: Results From an International Online Survey.

PURPOSE: To describe the scope of practice of physical therapists (PTs) working with children worldwide. METHODS: PTs working with children in any context and country were invited via social media and email campaigns to complete an online survey containing 42 questions about work context and service delivery. Descriptive statistics were computed. RESULTS: Of the 1133 participants from 77 countries, most worked with children full-time (51.8%), and in government-funded work settings (57.5%). Modalities of access to services varied across countries, work settings, and children's conditions, yet 46.7% of PTs reported that most children had direct access to services. PTs provided services to children with a variety of conditions, with cerebral palsy being most reported (83.3%). Interventions focused primarily on improving body function (42.0%) and on providing face-to-face individual treatment (96.6%). CONCLUSIONS: This study provides an international portrait of pediatric PT practice and illustrates the diversity of services in pediatric PT.

Implementing Partnering for Change in Québec: Occupational Therapy Activities and Stakeholders' Perceptions.

BACKGROUND.: The occupational therapy school-based Partnering for Change (P4C) model has mostly been documented in Ontario. PURPOSE.: This implementation study describes the implementation of P4C in two Québec elementary schools (P4C-Q), as well as therapy practices, their impacts, factors perceived to influence implementation, and recommendations. METHOD.: A sequential mixed-methods design was applied. Therapists (n=2) completed daily journals, describing activities by P4C-Q level. Therapists and other school-stakeholders (n=11) participated in semi-structured interviews, analyzed through a content analysis framework. FINDINGS.: Daily journals illustrated that the majority of therapy time was spent on activities targeting the entire classroom, and on collaboration with educators. Interviews illustrated how coaching was used across different practices and the impact of these practices for schools (e.g., capacity-building) and children (e.g., increased functioning), and highlighted how relationship-building is key to facilitating the implementation of this model. IMPLICATIONS.: Lessons learned may be helpful for others implementing P4C in their own contexts.

Preschoolers' Developmental Profiles and School-Readiness in a Low-Income Canadian City: A Cross-Sectional Survey.

A joint initiative between community and public health stakeholders in a low-income Canadian city was created to describe the developmental profiles of children aged 2-5 years. A two-phase, cross-sectional design was used. Children's development was assessed using an online screening procedure. Those at risk of delays were invited for a school-readiness face-to-face brief assessment. Descriptive and exploratory analyses were conducted. In Phase 1, 223 families were screened; 100 children were at risk of delays (45%); 13% were at risk in ≥3 developmental domains; 26% were at risk in the fine motor domain. Risk of delay was associated with parental concerns, accessing more healthcare professionals, and using fewer public health/community programs. Lower incomes, and not attending day care showed trends towards an increased risk of delay. In Phase 2, 49 children were assessed; 69% were at risk of school-readiness delays; 22% had potential motor delays; 37% were at risk in the social domain. This study found a higher proportion of children at risk of delay than typically reported. Creating community partnerships could help identify all children needing developmental and school-readiness support. More research is needed to ensure these community-based partnerships are integrated into health/community programs responding to children's needs and parental concerns.

Evaluation of a Web Platform Aiming to Support Parents Having a Child with Developmental Coordination Disorder: Brief Report.

Objective: To explore the effects of a web platform, aiming to support parents of children 5-12 years old with suspected or diagnosed developmental coordination disorder (DCD), on parental knowledge and skills.Method: A randomized pilot trial was undertaken (Clinical trial NCT03141333). Parents of the intervention group (n = 15) had access to a web platform (including resources, forum and virtual interactions) for three months. The control group (n = 13) only had access to resources. The primary outcome was measured pre- and post-intervention with the Parent Knowledge and Skills Questionnaire. Pre- post-questionnaires evaluated secondary outcomes (parents' sense of competence, children's strenghts and difficulties, and occupational performance).Results and discussion: All outcome measures improved over time for the intervention group. However, those improvements were not clinically or statistically significant (p 0.08-0.41).Conclusion: Web platforms supporting parents of children with DCD need further evaluation. Especially, usability of web platforms and new outcome measures should be explored.

Diversity of practices in telerehabilitation for children with disabilities and effective intervention characteristics: results from a systematic review.

Purpose: To describe the characteristics and effectiveness of pediatric telerehabilitation interventions offered to children 0-12 years old or to their families.Methods: A systematic review was conducted on randomized control trials published between 2007 and 2018 involving at least one rehabilitation professional who provided services remotely. Information was extracted about key study, participants and intervention characteristics. The percentage of outcomes that improved were computed per study, and per intervention characteristic.Results: Out of 4472 screened articles, 23 were included. Most studies were published after 2016 and evaluated outcomes related to the child's behavior (n = 12, 52.2%) or to the parent (n = 10, 43.5%), such as parental skills or stress. Overall, 56.1% (SD: 38.5%) of evaluated outcomes improved following telerehabilitation. A great diversity of population and teleintervention characteristics was observed. Effective interventions tended to target parents, centered around an exercise program, used a coaching approach, focused on improving children's behavioral functioning, lasted >8 weeks and were offered at least once a week.Conclusions: Intervention characteristics that appear to yield better outcomes should inform the development of future telerehabilitation studies, especially in populations for whom telerehabilitation is currently understudied (e.g., children's with physical functioning difficulties). Future trials should compare telerehabilitation interventions to well-described evidence-based face-to-face interventions, and document their cost-effectiveness.Implications for RehabilitationDespite a great variety in practices, telerehabilitation might be as effective as face-to-face interventions, across disciplines, for a variety of clinical outcomes.Telerehabilitation might be more effective when coaching approaches are used, especially to achieve outcomes related to children's behavior or parental skills.Further research is required to better understand the characteristics of effective telerehabilitation interventions, and to determine how these characteristics may differ for specific populations and outcomes.

Research and knowledge transfer priorities in developmental coordination disorder: Results from consultations with multiple stakeholders.

BACKGROUND: Priority-setting is a way to focus research and knowledge translation (KT) efforts for community-based research partnerships (CBRP). OBJECTIVE: To identify the developmental coordination disorder (DCD) research and KT priorities of stakeholders in Quebec, Canada, and their perceptions regarding the implementation of a CBRP. DESIGN: An advisory committee oversaw the research process including an online survey and four community forums. SETTING AND PARTICIPANTS: The survey was posted online and four community forums were organized. Participants included parents of children with DCD, adults with DCD, health professionals and school staff. MAIN VARIABLES: Stakeholder generated research and KT priorities, and optimal CBPR conditions. OUTCOME MEASURES: Participants selected their top five priorities based on a predefined list of 16 research and 12 KT priorities determined in collaboration with the advisory committee. They also rated the importance of various CBRP conditions. Preliminary survey results were discussed during the forums. RESULTS: Survey participants (n = 395) identified interwoven research and KT priorities where access to services was considered to be essential: supporting children at school; improving DCD identification and diagnosis; preventing secondary consequences; improving the organization of services and implementing effective services. Forum participants (n = 52) confirmed the relevance of these priorities and supported the establishment of a CBRP inclusive of all stakeholders to improve DCD services, research and KT. DISCUSSION AND CONCLUSIONS: A general consensus emerged among all groups, but adults with DCD were more concerned with employment than were the other stakeholder groups. These findings are presently being used to shape an ongoing, online CBRP.

Exploring engagement in a virtual community of practice in pediatric rehabilitation: who are non-users, lurkers, and posters?

PURPOSE: Communities of practice are increasingly recognized in rehabilitation as useful knowledge transfer tools; however, little is known about their users. This exploratory study describes the characteristics of participants and non-participants invited to engage in a pediatric rehabilitation virtual community of practice. In addition, we explored virtual community of practice utilization behaviors, engagement predictors, and the impact of strategies designed to foster engagement. MATERIALS AND METHODS: Participants' demographics including information-seeking style and organization e-readiness, as well as online platform frequency of use data were collected and analyzed using descriptive, comparative, and predictive statistics. RESULTS: Seventy-four percent of those invited used the virtual community of practice. Users had less years of experience in pediatric rehabilitation than non-users. Among the users, 71% were classified as "lurkers," who engaged through reading content only; while 29% were classified as "posters," editing online content. Predictive factors were not uncovered, however an increased number of forum visits correlated with being a poster, a non-information seeker, an employee of an organization demonstrating e-readiness, and regularly working with children with the virtual community of practice specific condition. User-engagement strategies increased visits to the forum. CONCLUSIONS: These findings will assist rehabilitation leaders in leveraging rehabilitation-specific virtual community of practice to improve knowledge transfer and practice in pediatric rehabilitation and disability management. Implications for Rehabilitation Communities of practice are increasingly recognized as useful knowledge transfer tools for rehabilitation professionals and are made more accessible thanks to virtual technologies. Our virtual community of practice was found to be optimized in health care organizations with an electronic culture, when the topic area had daily relevance to its target audience, and was particularly beneficial for those who have limited years of experience in pediatric rehabilitation. A strongly committed, selected leadership team with the technological skills, content expertise, and designated time to maintain the site and to nurture discussion was deemed vital in fostering knowledge exchange in this context. User-focused engagement strategies showed promise in increasing visits to the virtual community of practice. Our study supports the importance of multi-pronged approaches in enhancing health care professional knowledge and skills Findings from this study will assist rehabilitation leaders in optimally leveraging rehabilitation-specific virtual community of practice to improve knowledge transfer in pediatric rehabilitation and disability management.

Recruitment, use, and satisfaction with a web platform supporting families of children with suspected or diagnosed developmental coordination disorder: a randomized feasibility trial.

Objectives. To determine the feasibility of recruiting families of children with suspected or diagnosed developmental coordination disorder (sdDCD) and explore their satisfaction with a web-based intervention Design. A feasibility randomized trial was conducted. Participants were the parents of 5-12-year-old children with sdDCD. The intervention group had access to online resources, group and private forums, and videoconferencing with a therapist. Main outcomes were recruitement and retention rates. Satisfaction was documented through a post-intervention survey and interview. Results. The recruitment rate was seven participants per month (n = 28 participants) and retention rate was 68%. Satisfaction was moderate. No differences in use and satisfaction were observed between groups. Participants formulated recommendations for improving the intervention, including targeting families earlier in the diagnosis process, and pre-scheduling meetings with therapists. Conclusions. This study demonstrated the feasibility of future trials, and highlighted avenues for improvement. Parent involvement during the development of the intervention is discussed at length. Abbreviations: DCD: Developmental Coordination Disorder; sdDCD: suspected or diagnosed Developmental Coordination Disorder; RCT: randomized-controled trial.

New Reference Values for Cardiopulmonary Exercise Testing in Children.

INTRODUCTION: Cardiopulmonary exercise testing is an essential tool to assess cardiorespiratory fitness (CRF) in children. There is a paucity of adequate pediatric reference values that are independent of body size and pubertal stage. The purpose of this study is to provide Z score equations for several maximal and submaximal CRF parameters derived from a prospectively recruited sample of healthy children. METHODS: In this cross-sectional multicenter study, we prospectively recruited 228 healthy children 12 to 17 yr old in local schools. We performed a symptom-limited cardiopulmonary exercise testing progressive ramp protocol on an electronically braked cycle ergometer. Eighteen CRF parameters were analyzed. We tested several regression models to obtain prediction curves that minimized residual association with age, body size, and pubertal stage. Both the predicted mean and the predicted SD were modeled to account for heteroscedasticity. RESULTS: We identified nonlinear association of CRF parameters with body size and significant heteroscedasticity. To normalize CRF parameters, the use of a single body size variable was not sufficient. We therefore used multivariable models with various combination of height, corrected body mass, and age. Final prediction models yielded adjusted CRF parameters that were independent of age, sex, body mass, height, body mass index, and Tanner stages. CONCLUSIONS: We present Z score equations for several CRF parameters derived from a healthy pediatric population. These reference values provide updated predicted means and range of normality that are independent of sex and body size. Further testing is needed to assess if these reference values increase sensitivity and specificity to identify abnormal cardiorespiratory response in children with chronic diseases.

Developing a Web Platform to Support a Community of Practice: A Mixed Methods Study in Pediatric Physiotherapy.

INTRODUCTION: Web platforms are increasingly used to support virtual interactions between members of communities of practice (CoP). However, little is known about how to develop these platforms to support the implementation of best practices for health care professionals. The aim of this article is to explore pediatric physiotherapists' (PTs) perspectives regarding the utility and usability of the characteristic of a web platform developed to support virtual communities of practice (vCoP). METHODS: This study adopted an explanatory sequential mixed methods design. A web platform supporting the interactions of vCoP members was developed for PTs working with children with developmental coordination disorder. Specific strategies and features were created to support the effectiveness of the platform across three domains: social, information-quality, and system-quality factors. Quantitative data were collected from a cross-sectional survey (n = 41) after 5 months of access to the web platform. Descriptive statistics were calculated. Qualitative data were also collected from semistructured interviews (n = 9), which were coded, interpreted, and analyzed by using Boucher's Web Ergonomics Conceptual Framework. RESULTS: The utility of web platform characteristics targeting the three key domain factors were generally perceived positively by PTs. However, web platform usability issues were noted by PTs, including problems with navigation and information retrieval. DISCUSSION: Web platform aiming to support vCoP should be carefully developed to target potential users' needs. Whenever possible, users should co-construct the web platform with vCoP developers. Moreover, each of the developed characteristics (eg, newsletter, search function) should be evaluated in terms of utility and usability for the users.

Can a Community of Practice Improve Physical Therapists' Self-Perceived Practice in Developmental Coordination Disorder?

BACKGROUND: Communities of practice (CoPs) are useful knowledge translation (KT) strategies, but little is known about their impact on physical therapists' self-perceived practice. PURPOSE: The impact of a CoP on physical therapists' self-perceived practice was evaluated, and factors influencing changes in self-perceived knowledge, skills, and practice related to developmental coordination disorder (DCD) were explored. DESIGN: An explanatory sequential mixed-methods design was used, guided by the Theory of Reasoned Action and Theory of Planned Behavior. METHODS: Physical therapists participated in a DCD physical therapist CoP, which included 2 full-day, face-to-face workshops, with access to a 5-month online forum between the workshops, and completed questionnaires at 3 time-points: before the first workshop, before accessing the online forum, and following the second workshop. Measures completed before and after the CoP included closed-ended questions providing global scores on therapists' self-perceived knowledge, skills, and practice. Physical therapists' sociodemographic characteristics, information-seeking style, use of the online forum, and behavioral change goals were also collected. Paired t-tests, ANCOVAs, and linear regression models were used to analyze the data. RESULTS: Forty-one physical therapists completed all questionnaires. Their self-perceived knowledge, skills, and practice change scores were significantly higher (+0.47, +1.23, and +2.61, respectively; P < .001) at the end of the CoP compared with the beginning. Few of the factors explored significantly influenced therapists' self-reported change scores. LIMITATIONS: No observational data on practice change was collected. The small sample may have limited the ability to identify factors influencing self-perceived practice changes. CONCLUSIONS: The CoP increased physical therapists' self-perceived knowledge, skills, and practice. More research is needed to explore CoP impact on physical therapist practices and how behavioral changes influence patient outcomes.

Virtual Knowledge Brokering: Describing the Roles and Strategies Used by Knowledge Brokers in a Pediatric Physiotherapy Virtual Community of Practice.

INTRODUCTION: Knowledge transfer in pediatric rehabilitation is challenging and requires active, multifaceted strategies. The use of knowledge brokers (KBs) is one such strategy noted to promote clinician behavior change. The success of using KBs to transfer knowledge relies on their ability to adapt to ever-changing clinical contexts. In addition, with the rapid growth of online platforms as knowledge transfer forums, KBs must become effective in virtual environments. Although the role of KBs has been studied in various clinical contexts, their emerging role in specific online environments designed to support evidence-based behavior change has not yet been described. Our objective is to describe the roles of, and strategies used by, four KBs involved in a virtual community of practice to guide and inform future online KB interventions. METHODS: A descriptive design guided this study and a thematic content analysis process was used to analyze online KB postings. The Promoting Action on Research in Health Sciences knowledge transfer framework and online andragogical learning theories assisted in the coding. A thematic map was created illustrating the links between KBs' strategies and emerging roles in the virtual environment. RESULTS: We analyzed 95 posts and identified three roles: 1) context architect: promoting a respectful learning environment, 2) knowledge sharing promoter: building capacity, and 3) linkage creator: connecting research-to-practice. Strategies used by KBs reflected invitational, constructivism, and connectivism approaches, with roles and strategies changing over time. DISCUSSION: This study increases our understanding of the actions of KBs in virtual contexts to foster uptake of research evidence in pediatric physiotherapy. Our results provide valuable information about the knowledge and skills required by individuals to fulfill this role in virtual environments.

Using an evidence-based online module to improve parents' ability to support their child with Developmental Coordination Disorder.

BACKGROUND: Developmental Coordination Disorder (DCD) is a prevalent neurodevelopmental disorder. Best practices include raising parents' awareness and building capacity but few interventions incorporating these best practices are documented. OBJECTIVE: To examine whether an evidence-based online module can increase the perceived knowledge and skills of parents of children with DCD, and lead to behavioral changes when managing their child's health condition. METHODS: A mixed-methods, before-after design guided by the Theory of Planned Behavior was employed. Data about the knowledge, skills and behaviors of parents of children with DCD were collected using questionnaires prior to completing the module, immediately after, and three months later. Paired T-tests, sensitivity analyses and thematic analyses were performed on data as appropriate. RESULTS: One hundred-sixteen, 81 and 58 participants respectively completed the three questionnaires. For knowledge and skills, post- and follow-up scores were significantly higher than baseline scores (p < 0.01). Fifty-two (64%) participants reported an intention to change behavior post-intervention and 29 (50%) participants had tried recommended strategies at follow-up. Three themes emerged to describe parents' behavioral change: sharing information, trialing strategies and changing attitudes. Factors influencing parents' ability to implement these behavioral changes included clear recommendations, time, and 'right' attitude. Perceived outcomes associated with the parental behavioral changes involved improvement in well-being for the children at school, at home, and for the family as a whole. CONCLUSIONS: The online module increased parents' self-reported knowledge and skills in DCD management. Future research should explore its impacts on children's long-term outcomes.

A Systematic Review of Reference Values in Pediatric Cardiopulmonary Exercise Testing.

Cardiopulmonary exercise testing (CPET) is used for the diagnosis and prognosis of cardiovascular and pulmonary conditions in children and adolescents. Several authors have published reference values for pediatric CPET, but evaluation of their validity is lacking. The aim of this study was to review pediatric CPET references values published between 1980 and 2014. We specifically assessed the adequacy of the normalization methods used to adjust for body size. Articles that proposed references values were reviewed. We abstracted information on exercise protocols, CPET measurements and normalization methods. We then evaluated the studies' methodological quality and assessed them for potential biases. Thirty-four studies were included. We found important heterogeneity in the choice of exercise protocols and in the approach to adjustment for body size or other relevant confounding factors. Adjustment for body size was principally done using linear regression for age or weight. Assessment of potential biases (residual association, heteroscedasticity and departure from the normal distribution) was mentioned in only a minority of studies. Our study shows that contemporary pediatric reference values for CPET have been developed based on heterogeneous exercise protocols and variable normalization strategies. Furthermore, assessment of potential bias has been inconsistent and insufficiently described. High-quality reference values with adequate adjustment for confounding variables are needed in order to optimize CPET's specificity and sensitivity to detect abnormal cardiopulmonary response to exercise.

Economic evaluation of a 100% whey-based partially hydrolyzed infant formula in the prevention of atopic dermatitis among Danish children.

OBJECTIVE: A pharmacoeconomic analysis was undertaken to determine costs, consequences, and cost-effectiveness of a brand of partially hydrolyzed 100%-whey formula manufactured by Nestlé (PHF-W), in the prevention of atopic dermatitis (AD) in 'at risk' Danish children compared to extensively hydrolyzed formula (EHF-Whey or Casein). METHODS: Given the non-significant differences between PHF-W and EHF, the base case analytic approach amounted to a cost-minimization analysis (CMA) reporting the difference in formula acquisition costs over the period of formula consumption for the population of interest. However, sensitivity analyses (SAs) were undertaken to explore applying the nominal efficacy of PHF-W and EHF, thus leading to a cost-effectiveness analysis (CEA). Hence, an economic model based on a 12-month time horizon was developed synthesizing treatment pathways, resource utilization, and costs associated with the treatment of AD in the population of interest. The final economic outcome of the SAs was the incremental cost per avoided case (ICER) defined as the expected cost per avoided case of AD for PHF-W vs EHF, determined from three perspectives: the Ministry of Health (MOH), the family of the subject, and society (SOC). RESULTS: In the base case CMA, savings of DKK 9 M, DKK 20 M, and DKK 29 M were generated for PHF-W vs EHF from the MOH, family, and SOC perspectives. In the sensitivity CEA, PHF-W was dominant over EHF-Whey from all perspectives, while EHF-Casein displayed against PHF-W unattractive ICERs of DKK 315,930, DKK 408,407, and DKK 724,337 from the MOH, family, and SOC perspectives. Probabilistic SAs indicated that PHF-W was 86% likely to be dominant over EHF-Whey, whereas EHF-Casein had no likelihood of dominating PHF-W. CONCLUSION: Under a range of assumptions, this analysis demonstrated the attractiveness of PHF-W vs both types of EHF in the prevention of AD among 'at risk' Danish infants who are not or cannot be exclusively breastfed.

Methylnaltrexone in the treatment of opioid-induced constipation in cancer patients receiving palliative care: willingness-to-pay and cost-benefit analysis.

CONTEXT: When laxative regimens have failed, methylnaltrexone may be indicated for the relief of opioid-induced constipation (OIC) in patients with advanced illness receiving palliative care. OBJECTIVES: A cost-benefit analysis (CBA), based on a willingness-to-pay (WTP) approach, was performed to determine if methylnaltrexone should be added to the formulary list of drugs being reimbursed by third-party payers in Canada for the treatment of cancer patients in palliative care suffering from OIC. METHODS: The WTP study had two components: a decision board explaining treatment options (Component A) and a questionnaire to measure individual WTP using a bidding game approach (Component B). Component A had two options: Option 1 (laxatives only) and Option 2 (laxatives+methylnaltrexone injection). Only participants choosing Option 2 were invited to complete Component B. The results of the WTP survey were then incorporated into a CBA. Within a hypothetical cohort, additional monthly premiums that individuals were willing to pay for methylnaltrexone were compared with the monthly costs to the insurer for providing methylnaltrexone to all patients who would potentially be using it. RESULTS: Four hundred one Canadians, of age 18 years and older, were surveyed and yielded a WTP in additional monthly insurance premiums of Canadian dollar (CAD) $8.65 (95% confidence interval: CAD$6.17-CAD$11.13). The CBA resulted in additional CAD$89,307 with a cost of CAD$139,840 and benefits of CAD$229,147. A set of 10,000 Monte Carlo simulations resulted in average CBA savings of CAD$145,011 with a 99.86% probability of dominance. CONCLUSION: The present CBA provides pharmacoeconomic evidence for the adoption of methylnaltrexone for treating OIC in terminally ill cancer patients.

Meta-analysis of a partially hydrolysed 100%-whey infant formula vs. extensively hydrolysed infant formulas in the prevention of atopic dermatitis.

OBJECTIVES: This study presents previously unpublished point and cumulative incidence rates and relative risks (RRs) for comparing a partially hydrolysed 100% whey-based infant formula, NAN-HA * (PHF-W) to extensively hydrolysed whey- (EHF-Whey) or casein-based (EHF-Casein) infant formulas in the prevention of atopic dermatitis (AD) in infants who cannot be breastfed exclusively. It also outlines methods to convert the above-mentioned data as well as data comparing PHF-W to cows' milk formula (SF) into inputs to be applied to a pharmacoeconomic model. * NAN-HA is a registered trade name of Nestlé SA, Switzerland. METHODS: The incidence rates and RRs were obtained from a meta-analysis which analysed efficacy for PHF-W vs. EHF but did not present those. It took into consideration any relevant randomized controlled trial which compared the use of PHF-W with SF or EHF for the prevention of allergies. The primary outcomes of interest were the incidence, cumulative incidence and period prevalence of allergic manifestations and of AD in particular. Fifteen studies had been included for analysis of which six studies explored PHF-W vs. EHF. These results and PHF-W vs. SF data were adapted for inputs into a pharmacoeconomic model which used a spreadsheet decision-analytic economic model based on 3-month cycles to explore the cost-effectiveness of PHF-W vs. SF and EHF. Weights were applied to the incidence rates and RRs for each reported time period which were then adapted into 3-month indicators. RESULTS: This meta-analysis for PHF-W (557 patients) vs. EHF-Whey (559 patients) yielded RR of 0.75 (0.54, 1.05) and 0.80 (0.63, 1.02) at 0-12 months and at 0-36 months, respectively. Corresponding RRs for PHF-W vs. EHF-Casein (580 patients) were 1.06 (0.74, 1.53) at 0-12 months and 1.13 (0.87, 1.47) at 0-36 months. CONCLUSION: It appears that the efficacy of PHF-W falls within the range of that of both EHF formulas (whey and casein) and allows the application of these results in a pharmacoeconomic model.